Abstract

SummaryClinical trials are crucial for drug development but often face uncertain outcomes due to safety, efficacy, or patient-recruitment problems. We propose the Hierarchical Interaction Network (HINT) to predict clinical trial outcomes. First, HINT encodes multi-modal data (drug molecule, target disease, trial eligibility criteria) into embeddings. Then, HINT trains knowledge-embedding modules using drug pharmacokinetic and historical trial data. Finally, a hierarchical interaction graph connects all of the embeddings to capture their interactions and predict trial outcomes. HINT was trained and validated on 1,160 phase I trials, 4,449 phase II trials, and 3,436 phase III trials. It obtained 0.665, 0.620, and 0.847 F1 scores on separate test sets of 627 phase I, 1,653 phase II, and 1,140 phase III trials, respectively. HINT significantly outperforms the best baseline method on most metrics. The benchmark dataset and codes are released at https://github.com/futianfan/clinical-trial-outcome-prediction.

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